Today, we’re outlining key concepts from the 2026 American College of Cardiology (ACC) scientific statement, Gene Editing Therapy in Cardiovascular Disease. This scientific statement provides an overview of emerging gene editing technologies and their potential role in transforming cardiovascular care, and includes basic science supporting gene editing, the types of cardiovascular diseases most amenable for initial application of gene editing, and the challenges associated with gene editing. 

Below, we have key concepts from the scientific statement. Refer to the full-text version of the scientific statement for the complete overview. 

Key Concepts from the 2026 Scientific Statement

Current Landscape of Gene Editing Therapies in Cardiovascular Diseases 

The 2026 ACC scientific statement on gene editing therapy highlights the role of gene editing therapies in several cardiovascular diseases, including transthyretin amyloid cardiomyopathy (ATTR-CM), lipid disorders, myocardial diseases such as Duchenne muscular dystrophy, hypertrophic cardiomyopathy, and dilated cardiomyopathy, as well as vascular conditions, such as syndromic aortopathies.

Key advances of gene editing therapies include:

  • Improved understanding of cardiovascular disease (CVD) genetics
  • Clustered regularly interspaced short palindromic repeats (CRISPR)–associated protein 9 (Cas9) breakthroughs in gene editing precision
  • Recognition of gene editing therapy hepatic treatment targets in transthyretin (TTR) amyloidosis and hyperlipidemia
  • Lipid nanoparticle (LNP) delivery to hepatocytes have minimal off-target effects

Challenges of gene editing therapy include:

  • Many CVDs have complex genetics that are not amenable to a single gene editing therapy
  • Myocardial delivery sites require viral delivery with associated risks
  • Risk of off-target effects and germline transmission
  • Unique clinical trial need, including prolonged safety follow-up

Challenges in the Clinical Utilization and Application of Gene Editing Therapy

Many CVDs have a genetic basis, but this is often under-recognized due to complex inheritance pattern, variable expressivity, reduced disease penetrance, and overlap with non-genetic forms of disease. Although guidelines recommend genetic testing for many inherited cardiovascular diseases, it remains poorly utilized, with only a small percentage of patients receiving appropriate testing. Barriers include limited provider awareness, referral access to genetic specialists and testing, and variability in institutional and payor support. Additionally, cascade testing for asymptomatic family members remains underutilized due to concerns regarding patient privacy, higher premiums, and the potential for increased health anxiety. There is also a need for clearer guidance on broader genomic screening and the identification of pathogenic cardiovascular disease variants. 

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